Summary
On September 17, 2026, the FDA approved Ultragenyx’s FAYUVI (rebisufligene etisparvovec-hopf) as the first treatment targeting the underlying cause of Sanfilippo syndrome type A in pediatric patients with preserved neurodevelopmental function.
Data from the pivotal Transpher A study and long-term follow-up showed sustained reductions in cerebrospinal fluid heparan sulfate and higher cognitive scores versus an external natural-history cohort.
FAYUVI was rejected by the FDA in July 2025 because of manufacturing issues involving Ultragenyx’s facility and a third-party site. After resubmission and priority review, the therapy received full approval in Se...
